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BioWorld - Thursday, May 8, 2025
Breaking News: 100 days of uncertaintyBreaking News: 100 days of uncertaintyBreaking News: 100 days of uncertaintyBreaking News: 100 days of uncertainty
  • DNA and silhouette

    Organoids plus gene editing bring insights into pediatric metabolic disease

    “I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
  • ABI-201 confers protection against age-related macular degeneration

  • Viverita and Boehringer Ingelheim collaborate on cancer target discovery and validation

  • Organoids plus gene editing bring insights into pediatric metabolic disease

    “I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
  • ABI-201 confers protection against age-related macular degeneration

    ABI-201 is an engineered AAV.N54 vector that expresses a soluble CD59 variant and a complement 3 (C3) inhibitor fusion protein. ABI-201 was developed by Avirmax Biopharma Inc. to confer photoreceptor protection against retinal damage. The effects of ABI-201 were tested in a sodium iodate-induced retinal damage model in nonhuman primates, a widely used model for testing therapies for age-related macular degeneration (AMD).
  • Viverita and Boehringer Ingelheim collaborate on cancer target discovery and validation

    Viverita Therapeutics Inc. has established a strategic research collaboration with Boehringer Ingelheim Pharma GmbH & Co. KG aimed at accelerating the discovery and validation of novel therapeutic targets for cancer.
  • WTX-921: An IL-10 Indukine designed to minimize systemic toxicity

    Interleukin-10 (IL-10) is an immunoregulatory cytokine produced by a variety of immune cells, including type 1 regulatory T cells and macrophages, as well as nonimmune cells such as epithelial cells and fibroblasts.
  • Merck Sharp & Dohme patents new IL4I1 inhibitors

    Merck Sharp & Dohme LLC has disclosed L-amino acid oxidase (IL4I1; LAAO) inhibitors reported to be useful for the treatment of cancer.
  • Genprex and University of Pittsburgh sign new agreement to study GPX-002 for diabetes

    Genprex Inc. and University of Pittsburgh have entered into a new sponsored research agreement (SRA) to study GPX-002, Genprex’s gene therapy for diabetes in animal models of type 1 and type 2 diabetes.
  • Preclinical results from a new HPV16/18 mRNA-based vaccine presented

    Metis Pharmaceuticals Inc. recently presented a novel mRNA-encoded human papillomavirus (HPV) 16/18 therapeutic vaccine for HPV-driven malignancies.
  • Two cores better than one: combining carboline and triazole into small molecules against malaria

    Illustration of blood cells, Plasmodium causing malaria illness
  • ETH Zürich and Roche divulge new MAGL inhibitors

  • Bristol Myers Squibb describes new GSPT1 degradation inducers

  • Blocking GAPDH aggregation to mitigate stroke injury

    Cross-section of brain
  • ADCE-T02 ADC against tissue factor shows strong antitumor activity

  • Sorafenib analogues disclosed in University of Barcelona patent

  • SLC25A19 predicts prognosis in hepatocellular carcinoma

    Photomicrograph of hepatocellular carcinoma
  • Tenvie Therapeutics discovers new TMEM175 modulators

BioWorld Insider Podcast

Play buttonRobert Williamson, CEO of Triumvira Immunologics, and Hernan Bazan, CEO of South Rampart Pharma, discuss bracing for tariffs, NIH grant cuts and gifting China a biopharma leadership position.
Listen now

Conferences

  • 3D illustration of human digestive system

    LM-350 shows strong antitumor activity in CDH17-positive models

    New compound
    Cadherin 17 (CDH17) is a membrane-bound cell adhesion molecule involved in tumor cell proliferation and is selectively overexpressed in several gastrointestinal malignancies, including colorectal cancer, gastric cancer and pancreatic cancer.
  • Drug R&D concept image.

    Surrozen’s SZN-8141 ameliorates retinopathy in murine models

    Ocular
    Recent evidence has suggested that the use of norrin mimetics targeting both frizzled-4 (FZD4) and low-density lipoprotein receptor-related protein 5 (LRP5) may be highly effective at modulating retinal vascular leakage. When combined with an anti-VEGF therapy, it was hypothesized to have an...
  • Beigene’s BGB-A3055 inhibits tumor growth in cancer rodent models

    American Association for Cancer Research
    Beigene Co. Ltd. has presented data on their anti-CCR8 antibody BGB-A3055 as a cancer immunotherapeutic approach.
  • PBI-671 induces retinal ganglion cell survival in glaucoma models

    Ocular
    Glaucoma is a leading cause of blindness and is characterized by the death of retinal ganglion cells (RGCs). Genetic screening data have identified dual leucine zipper kinase (DLK) and leucine zipper kinase (LZK) as important mediators of RGC death.
More in Conferences

Today's news in brief

  • Other news to note for May 7, 2025

  • Respiratory

  • Dermatologic

  • Cardiovascular

  • Hematologic

  • Nephrology

Patents

  • Canwell Biotech discovers new SHP-2 inhibitors

  • New HIV integrase inhibitors disclosed in Gilead patent

  • Korea Research Institute of Chemical Technology describes new glutaminase inhibitors

  • Chinese scientists divulge new IL-17A inhibitors

  • Korean researchers patent new autotaxin inhibitors

  • US researchers discover new phosphonamidate prodrugs

  • New lipid prodrugs of bromolysergide disclosed in Seaport Therapeutics patent

  • Jiangsu Simcere Pharmaceutical describes new IRAK-4 degradation inducers

  • Nutshell Therapeutics divulges new TP53 Y220C reactivators

  • Yuhan patents new HPK1 inhibitors

Cancer

  • Antibody-drug conjugate

    Formosa and Almac Discovery sign license agreement for EGFRxROR1 bispecific ADC

    License
    Formosa Pharmaceuticals Inc. and Almac Discovery Ltd. have announced a global licensing agreement for development and commercialization of ALM-401, a first-in-class engineered bispecific antibody-drug conjugate (ADC) for the treatment of solid tumors characterized by the dual expression of EGFR and...
  • Researchers develop potent and selective SGK1 inhibitors for prostate cancer treatment

  • New study unveils the potential oncogenic role of PPP2R1A in gastric cancer

  • Xenothera details preclinical characterization of XON-9

    Conferences
  • Optimized ligands for mitochondrial DNA-targeted phototherapy for triple-negative breast cancer

More in Cancer

Infection

  • Tiger mosquito

    Immunogenicity and efficacy of a bivalent self-amplifying RNA vaccine against yellow fever and Zika

    Immune
    Researchers from the Access to Advanced Health Institute and collaborating institutions have developed a promising new bivalent vaccine candidate that can protect against both yellow fever and Zika viruses. The study demonstrates that the vaccine elicits robust humoral and cellular immune responses...
  • Key genes and educated natural killer cells keep HIV in remission

    Immune
    Scientists at Institut Pasteur have gained new insights into how some people control HIV-1 replication after interruption of antiretroviral treatment (ART). The investigators found a fingerprint involved in long-term viral remission.
  • Nuvobio divulges new peptides to treat C. neoformans infection

    Patents
    Nuvobio Corp. has synthesized peptides reported to be useful for the treatment of Cryptococcus neoformans infection.
  • Ciprofloxacin prodrug effectively disrupts biofilm formation

  • GS-1 prevents varicella-zoster and herpes simplex virus entry

  • Harvard fighting back with lawsuit against Trump administration

    Science
  • Crestone’s CRS-700159 shows antiviral activity against coronaviruses

    Conferences
More in Infection

Neurology/psychiatric

  • Illustration of muscle tissue anatomy

    Solve FSHD supports Armatus Bio drug for facioscapulohumeral muscular dystrophy

    Financings
    Armatus Bio Inc.’s development of ARM-201, an AAV-delivered microRNA therapy for facioscapulohumeral muscular dystrophy (FSHD), has been boosted by a $3 million investment by Solve FSHD.
  • Conceptual 3D illustration showing degradation of motor neuron

    Alchemab and Lilly sign licensing agreement for ATLX-1282

    Deals and M&A
    Alchemab Therapeutics Ltd. has entered into a licensing agreement with Eli Lilly and Co. for ATLX-1282, Alchemab’s first-in-class IND-ready program targeting a novel receptor and mechanism for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.
  • Scientist looking in microscope, chemical structure concept image

    A SUMO1 peptide that might stop α-synuclein aggregation before it begins

    Peptide
    Abnormal aggregation of α-synuclein is thought to contribute to the pathology of Parkinson’s disease, the second most frequent neurodegenerative disorder. Therefore, developing drugs that prevent such aggregation could be effective for slowing or even preventing the disease.
  • New tachykinin NK3 receptor antagonists disclosed in Kallyope patent

    Patents
    Kallyope has divulged compounds acting as tachykinin NK3 receptor antagonists reported to be useful for the treatment of migraine.
  • Brain and DNA

    Unravel seeks Colombian approval to study RVL-001 for Rett and Pitt-Hopkins syndromes

    Regulatory
    Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.
More in Neurology/psychiatric

Immune

  • Seasonal flu vaccine found to trigger a broadly protective antibody targeting neuraminidase

  • Psychedelics modulate neuroimmune interactions that control fear

  • Granite Bio emerges from stealth with focus on antibodies for inflammatory, autoimmune and fibrotic conditions

  • Repertoire and Genentech partner on T cell-targeted therapies for an autoimmune disease

  • Poly-epitope influenza vaccine candidate triggers T-cell immunity across strains

  • Nanobody against mpox virus: from screening to crystal structure

  • Aclaris gains IND clearance for bispecific anti-TSLP/IL-4R antibody

  • IDOR-1117-2520 shows promise in the treatment of skin autoimmune diseases

  • WHO member states outline pandemic plan

  • Preclinical data on new composite peptide vaccine from Longhorn

Endocrine/metabolic

  • Test tube, dropper, DNA illustration

    Mammoth nominates development candidate for FCS and severe hypertriglyceridemia

    Cardiovascular
    Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.
  • Lack of NAD does not alter muscle function or accelerate aging

    Aging
  • Innorna’s mRNA therapy granted US orphan drug designation for Wilson disease

    Regulatory
  • Stem cell-derived liver organoids as a model of fatty liver disease

    Drug design, drug delivery and technologies
  • PIKfyve enzyme is target to ‘starve’ pancreatic cancer cells

    Science
More in Endocrine/metabolic

Biomarkers

  • Brain and DNA

    Variants in MED16 gene cause intellectual disability MEDopathy

    The Mediator complex is a system that regulates protein-coding gene transcription, where mediator of RNA polymerase II transcription subunit 16 (MED16) is a subunit belonging to this system. Pathogenic genetic variants in Mediator complex subunits usually lead to neurodevelopmental and...
  • Plasma ANG2 predicts poor outcomes after traumatic brain injury

    Conferences
    Investigators from the University of Pennsylvania have presented data regarding the relationship of angiopoietin-2 (ANG2) and its prognostic impact on traumatic brain injury (TBI). The ANG2 plasmatic levels were measured in patients with TBI (n=362), orthopedic injury controls (n=89) and healthy...
  • Ignite Biomedical, Liquid Biosciences discover new biomarkers for substance use disorder

    Substance use and poisoning
    Ignite Biomedical Inc. and Liquid Biosciences have announced the discovery of biomarkers to diagnose substance use disorder (SUD). The set of biomarkers includes some that have not previously been associated with addiction and spans all 6 SUDs (opioid, cocaine, methamphetamine, alcohol, nicotine...
  • CSPG4 expression discerns EBSCC from conventional ESCC

    Conferences
  • L1CAM predicts unfavorable outcome in malignant melanoma

    Conferences
  • INSM1 is highly sensitive marker in primary and metastatic Merkel cell carcinoma

    Conferences
  • NIPAL4 correlates with poor survival in renal cancer

    Cancer
More in Biomarkers

Gastrointestinal

  • Plant compound PEITC may mitigate both oxidative stress and inflammation in ulcerative colitis

    Ulcerative colitis, one type of chronic inflammatory bowel disease, is driven by a form of programmed cell death called pyroptosis.
  • Equillium advancing new AhR modulator

    Inflammatory
    Equillium Inc. is advancing EQ-504, a potent aryl hydrocarbon receptor (AhR) modulator acquired through the acquisition of Ariagen Inc., a company that was majority owned by Equillium’s largest investor, Decheng Capital.
  • Bristol Myers Squibb divulges new RIPK1 inhibitors

    Patents
    Bristol Myers Squibb Co. has synthesized receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of inflammatory bowel disease, psoriasis, rheumatoid arthritis, nonalcoholic or...
  • CREG1 protects against acetaminophen-induced liver damage by regulating autophagy

    Substance use and poisoning
    Acetaminophen (APAP), although recognized as a safe and effective antipyretic and analgesic drug, can lead to acute liver failure if taken in excess.
  • PVT-401 achieves milestone under Parvus and Abbvie inflammatory bowel disease collaboration

    Nanotech
    Parvus Therapeutics Inc. has announced the achievement of a milestone under its collaboration with Abbvie Inc. to develop treatments for inflammatory bowel disease (IBD). PVT-401, a novel peptide-major histocompatibility complex (pMHC)...
  • Germline variants’ impact on pan-cancer proteome

    Omics
    A large-scale study has revealed the impact of germline variants on proteins in 10 cancer types. Scientists from the National Cancer Institute’s Clinical Proteomic Tumor Analysis Consortium (CPTAC) conducted a precision proteogenomic analysis in a...
More in Gastrointestinal

BioWorld Insider Podcast

One-on-one with medical innovators

podcast microphone, sound waves on purple backgroundBreakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.


Recent episodes:

  • Two CEOs discuss bracing for tariffs, NIH grant cuts and
    gifting China biopharma leadership position
  • As biopharma investments stumble, med tech surges
  • Mega money well spent? Mammoth mergers fall short of the dream
  • Healing the health divide for women
  • Modifi gets creative on the rocky road to a multibillion-dollar buyout
  • A quantum leap into the future of drug development
  • Gene and cell therapies will propel innovation, says Astellas CCO
  • Capricor’s CEO pursues a BLA and talks rare disease
  • The first half of 2024 was strong as M&As and financings dominate
  • The cost of delays in development and sales: It’s probably not what you think

View all

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